Akcea and Ionis report positive results for Tegsedi in hATTR

Ionis Pharmaceuticals’ (Nasdaq: IONS) majority-owned subsidiary Akcea Therapeutics (Nasdaq: AKCA), whose shares were up 3.7% at $15.64 by midday in the USA, today announced publication of long-term data from the open-label extension (OLE) of the pivotal NEURO-TTR study of Tegsedi (inotersen) in patients with hereditary amyloidosis transthyretin (hATTR) with polyneuropathy.

The primary objective of the OLE study was to evaluate the safety and tolerability of long-term dosing with Tegsedi. Secondary objectives of the study included understanding progression based on measures such as the modified Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). Understanding changes over time in generic health-related quality of life based on the Short Form 36 Health Survey (SF-36) was an exploratory objective.

This interim analysis, published in the European Journal of Neurology, show that treatment with Tegsedi was not associated with additional safety concerns or signs of increased toxicity in study participants treated for up to five years. Treatment with Tegsedi resulted in continued efficacy in patients after two years. Results also showed that patients who started treatment with Tegsedi earlier (received Tegsedi treatment in the NEURO-TTR study) achieved greater long-term disease stabilization compared to those who switched from placebo to Tegsedi in the OLE study.